JoVE Logo
Faculty Resource Center

Sign In

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

DOI :

10.3791/57530-v

May 11th, 2018

May 11th, 2018

12,706 Views

1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Muscular Dystrophy Canada Research Chair, Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry

Various antisense oligonucleotides (AONs) have been shown to induce exon inclusion (splice modulation) and rescue SMN expression for spinal muscular atrophy (SMA). Here, we describe a protocol for AON lipotransfection to induce exon inclusion in the SMN2 gene and the evaluation methods to determine the efficacy in SMA patient fibroblasts.

Tags

Exon Inclusion

-- Views

Related Videos

article

Vascular Occlusion Training for Inclusion Body Myositis: A Novel Therapeutic Approach

article

Experimental Generation of Carcinoma-Associated Fibroblasts (CAFs) from Human Mammary Fibroblasts

article

Mouse Model of Intraluminal MCAO: Cerebral Infarct Evaluation by Cresyl Violet Staining

article

Cardiac Stress Test Induced by Dobutamine and Monitored by Cardiac Catheterization in Mice

article

Evaluation of Lung Metastasis in Mouse Mammary Tumor Models by Quantitative Real-time PCR

article

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

article

A Model of Glaucoma Induced by Circumlimbal Suture in Rats and Mice

article

Quantitative [18F]-Naf-PET-MRI Analysis for the Evaluation of Dynamic Bone Turnover in a Patient with Facetogenic Low Back Pain

article

Invasive Hemodynamic Assessment for the Right Ventricular System and Hypoxia-Induced Pulmonary Arterial Hypertension in Mice

article

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

JoVE Logo

Privacy

Terms of Use

Policies

Research

Education

ABOUT JoVE

Copyright © 2024 MyJoVE Corporation. All rights reserved